Amylyx Pharmaceuticals Inc., a Cambridge, MA-based developer of a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases, raised $5m in Series A Funding.
The round was led by Morningside Venture with participation from the ALS Investment Fund and former Genzyme CEO Henri Termeer, as well as new and previous private investors. In connection with the financing, Dr. Isaac Cheng, M.D. from Morningside and Mr. Felix von Coerper from the ALS Investment Fund have joined Amylyx’s board of directors.
The company intends to use the funds – which added to a recently awarded $2.96m grant to support the clinical trial from the ALS Accelerated Therapeutics Initiative – to conduct a Phase II Clinical Trial of AMX0035 for treatment of Amyotrophic Lateral Sclerosis.
Led by President Justin Klee and CEO Josh Cohen, Amylyx Pharmaceuticals About Amylyx Pharmaceuticals is developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases that target the neuroinflammation and nerve cell death that characterize these disorders. The company’s therapeutic candidate, AMX0035, is a proprietary, fixed-dose combination therapy that blocks nerve cell death and neurotoxic inflammation.
AMX0035 combines two drugs, sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), that Amylyx has shown act synergistically in preclinical models of ALS. Previous studies with PB and TUDCA as single agents demonstrated efficacy in several cellular and animal models of ALS. In addition, PB and TUDCA have been individually tested in clinical trials of ALS and both showed safety and tolerability and preliminary signs of efficacy.
The Phase II clinical trial will test the safety and tolerability of AMX0035, as well as functional outcomes.